Scientists in the US reacted to news of the Huntington’s breakthrough by doing a jig. (JONATHAN NACKSTRAND/AFP/Getty Images)
Huntington’s is one the most devastating diseases we can get with some patients describing it as Parkinson’s, Alzheimer’s and motor neurone disease all rolled into one. It is therefore not surprising that scientists reacted with some excitement as news emerged of “potentially the biggest breakthrough in neurodegenerative disease in the past 50 years”, according to one academic.
Researchers at University College London have shown that an experimental drug successfully lowers the harmful huntingtin protein – which is vital for brain development – in spinal fluid. It is the first time any drug has been shown to suppress the effects of the Huntington’s mutation that causes irreversible damage to the brain. Current treatments only help with symptoms, rather than slowing the disease’s progression.
Complex process
Finding a cure for any disease is a complex process.
The first goal has to be ensuring any potential drug does not have toxic side effects. Therefore, the main aim of the UCL study was determining whether the experimental drug – given to the majority of the 46 men and women from the UK, Germany and Canada with early stages Huntington’s who took part – is safe.
Around a quarter were given a placebo. The results showed the drug lowered the level of the toxic disease-causing protein in the nervous system and did so safely.
Anyone wondering just how excited scientists were should read the account by Dr Jeff Carroll, a neuroscientist at Western Washington University, on the HD Buzz website. “Both editors indulged in a little bit of happy dancing when they saw the press release,” he said.
“It’s really big news!”
Another reason for excitement is that the drug could not only be used to cure Huntington’s disease – though doctors stressed a much bigger and longer trial needs to take place before they are close to making that announcement – but may also help prevent other neurodegenerative diseases as well, such as Alzheimer’s and Parkinson’s.
https://inews.co.uk/essentials/news/science/huntingtons-disease-scientists-excited-game-changing-breakthrough/
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